HIV remission after heterozygous CCR5Δ32 stem cell transplant
2025 has been the most challenging year in the efforts to fight HIV since at least the advent of antiretroviral therapy. In a report on “Overcoming disruption, transforming the AIDS response,” released last week ahead of World AIDS Day on Dec. 1, the Joint United Nations Program on HIV/AIDS (UNAIDS) described “a global system in shock” by sharply reduced funding from the U.S. and other wealthy nations. Scientifically, for now, progress is ongoing. To mark World AIDS Day, Nature published three independent studies on HIV.

HIV remission after heterozygous CCR5Δ32 stem cell transplant
2025 has been the most challenging year in the efforts to fight HIV since at least the advent of antiretroviral therapy. In a report on “Overcoming disruption, transforming the AIDS response,” released last week ahead of World AIDS Day on Dec. 1, the Joint United Nations Program on HIV/AIDS (UNAIDS) described “a global system in shock” by sharply reduced funding from the U.S. and other wealthy nations. Scientifically, for now, progress is ongoing. To mark World AIDS Day, Nature published three independent studies on HIV.
XRD-0394 exhibits efficacy against glioma in vitro
High-grade gliomas, including glioblastomas, are aggressive cancers of the brain that usually recur despite standard treatment, including radiation therapy. These mechanisms conferring persistence to tumor cells may be explained by DNA damage response pathways, especially those relying on ataxia-telangiectasia mutated (ATM) and DNA-dependent protein kinase (DNA-PK), which induce the repair of radiation-induced DNA damage. The effects of the ATM/DNA-PK inhibitor XRD-0394 were evaluated in in vitro models of glioma.
Ascletis selects ASC-37 as development candidate for obesity
Ascletis Pharma Inc. has selected ASC-37 oral tablets as a clinical development candidate for the treatment of obesity. The company expects to submit an IND application to the FDA in the second quarter of next year.
Regeneron and Tessera collaborate on TSRA-196
Regeneron Pharmaceuticals Inc. and Tessera Therapeutics Inc. have established a global collaboration to develop and commercialize TSRA-196, Tessera’s lead investigational in vivo gene editing program for the treatment of α-1 antitrypsin deficiency (AATD).
STK-012 boosts T-cell activation while minimizing side effects
Researchers from Synthekine Inc. presented preclinical efficacy data on STK-012, a first-in-class IL-2 partial agonist engineered for preferential binding to the IL-2Rαβγ receptor.
Ideaya’s IDE-034 gains IND clearance for solid tumors
Ideaya Biosciences Inc. has received IND clearance from the FDA for the initiation of a phase I trial to evaluate IDE-034, a bispecific B7H3/PTK7 TOP1 antibody-drug conjugate.
Shenzhen Icarbonx Intelligent Peptide Pharmaceutical patents new peptides targeting BRAG2
Shenzhen Icarbonx Intelligent Peptide Pharmaceutical Technology Co. Ltd. has disclosed peptides targeting IQ motif and SEC7 domain-containing protein 1 (IQSEC1; BRAG2) reported to be useful for the treatment of stroke, major depression, substance abuse and dependence, and Alzheimer’s disease.

Conferences

HCW11-018 shows antitumor efficacy in pancreatic cancer
Society for Immunotherapy of Cancer
Pancreatic cancer is among the most aggressive cancer types, with survival rates being very low and current treatment being quite ineffective. To address this unmet medical need, HCW Biologics Inc. has developed and presented preclinical data for their T-cell engager approach – HCW11-018.
M-3554 exerts efficacy in soft tissue sarcoma
Cancer
M-3554 is an antibody-drug conjugate (ADC) targeting GD2; it has shown strong antitumor activity in neuroblastoma xenograft models and has been engineered to reduce anti-GD2 antibody-associated pain.
VT-3989 inhibits aggressive NF2-deficient tumor growth
Cancer
Vivace Therapeutics Inc. reported preclinical efficacy data of VT-3989, a TEAD inhibitor, in in vitro and in vivo models of aggressive meningioma. VT-3989 is in early clinical development for the treatment of patients with advanced solid tumors.
Next-generation PRMT5 inhibitor drives strong antitumor activity
Cancer
Researchers from Tango Therapeutics Inc. presented preclinical efficacy data on TNG-456, a selective MTA-cooperative PRMT5 inhibitor under development for the treatment of glioma and other tumors that frequently metastasize to the brain, such as non-small-cell lung cancer.

Cancer

New targets added to Fimecs and Astellas research
Degradation inducer
Raqualia Pharma Inc.’s Fimecs Inc. subsidiary has agreed with Astellas Pharma Inc. to add two new targets under their ongoing joint research. They entered into an agreement in 2022 to conduct joint research on targeted protein degradation.

Infection

Aerolysin-producing Aeromonas is behind ulcerative colitis
Gastrointestinal
Researchers from the Medical School of Nanjing University hypothesized that in ulcerative colitis, the gut-resident macrophages may be compromised, leading to impaired integrity of the epithelial barrier. “From a basic science standpoint, our work uncovers a novel etiology of ulcerative colitis....

Harmane-based quaternary ammonium salt against drug-resistant S. aureus
Researchers at Shandong Second Medical University have developed a dimethyl quaternary ammonium derivative of harmane that shows strong potential for attacking the bacterium through diverse mechanisms.
Arrepath describes new LpxH inhibitors
Patents
Arrepath Inc. has identified UDP-2,3-diacylglucosamine hydrolase (LpxH) (bacterial) inhibitors reported to be useful for the treatment of gram-negative bacterial infections.

Neurology/psychiatric

Derivatized TRPM2 inhibitor to more safely treat ischemic stroke
Inhibiting transient receptor potential melastatin 2 (TRPM2) may mitigate injury arising from ischemic stroke. Researchers at Zhejiang University and its medical school previously reported a small-molecule inhibitor of TRPM2, which also inhibited the human ether-a-go-go-related gene (hERG) channel,...
Epigenetic technology could eliminate misfolded prion proteins
Conferences
The number of deaths caused by prion diseases reaches about 30,000 annually. Only 5 months pass from the diagnosis of seemingly healthy patients to the fatal outcome of this neurodegenerative condition, and just 1 month until quality of life is completely lost. Removing the brain protein that...
Biohaven Therapeutics divulges new PKM2 activators
Patents
Biohaven Therapeutics Ltd. has synthesized pyruvate kinase M2 (PKM2) activators reported to be useful for the treatment of age-related macular degeneration, amyotrophic lateral sclerosis, multiple sclerosis, retinitis pigmentosa and fibrosis.
Chinese scientists discover new NMDA receptor antagonists
Patents
Researchers at Shanghai Institute of Materia Medica of the Chinese Academy of Sciences and Suzhou Vigonvita Life Sciences Co. Ltd. have described NMDA receptor antagonists and/or monoamine transporter inhibitors reported to be useful for the treatment of neurological disorders.
Captor Therapeutics patents new Nek7 degradation inducers
Degradation inducer
Captor Therapeutics Inc. has disclosed molecular glue degraders comprising a cereblon (CRBN) binding moiety acting as serine/threonine-protein kinase Nek7 degradation inducers reported to be useful for the treatment of amyotrophic lateral sclerosis, Still’s disease, Behçet’s disease, familial...

Endocrine/metabolic

Kedrion’s aceruloplasminemia treatment designated EU orphan drug
Regulatory
Kedrion SpA’s investigational plasma-derived treatment for congenital aceruloplasminemia has been awarded European orphan drug designation by the EMA. The company is working to advance this treatment toward clinical development in Europe.

Biomarkers

Mesothelin is biomarker, potential target in arthritic bone damage
Musculoskeletal
In a recent study published in Cell Reports Medicine, researchers from the Institute of Chinese Materia Medica of the China Academy of Chinese Medical Sciences and Xi’an Jiao Tong University Health Science Center found that Mesothelin (MSLN) was significantly elevated in rheumatoid arthritis (RA)...

UK Biobank delivers largest metabolomics dataset of 500K profiles
Science
It’s the biological resource that keeps on giving, and now UK Biobank has released the final tranche of data on the levels of 249 metabolites in the blood of its half a million participants.
With transdiagnostic approach, smaller target groups aren’t inevitably smaller markets
Neurology/psychiatric
Despite the formidable challenges for developing precision psychiatry, the approach is notching its first successes in the preclinical and even some clinical settings. Many individual studies as well as large projects like the Psychiatric Ratings using Intermediate Markers (PRISM) studies and the...

Gastrointestinal

Tangram seeks clinical trial clearance for TGM-312 for MASH
Regulatory
Tangram Therapeutics plc has submitted a clinical trial application (CTA) to the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II trial of TGM-312 for metabolic dysfunction-associated steatohepatitis (MASH).
Opko’s OPK-88006 shows hepatoprotection in MASH
Conferences
Opko Health Inc. has recently presented data for their GLP-1/glucagon receptor dual agonist OPK-88006, which is in preclinical development for the treatment of metabolic disease, including metabolic dysfunction-associated steatohepatitis (MASH)...
UK study reveals inconsistencies in global microbiome research
Regulatory
The U.K. Medicines and Healthcare products Regulatory Agency is calling for unified standards to harmonize microbiome research, after revealing major inconsistencies in the results when labs around the world analyzed identical reference samples of...
Samjin Pharmaceutical patents new compounds for MASH
Patents
Samjin Pharmaceutical Co. Ltd. has disclosed inhibitors of 17-β-hydroxysteroid dehydrogenase 13 (HSD17B13; 17-β-HSD 13) and/or estradiol 17-β-dehydrogenase 1 (HSD17B1; 17β-HSD1) and/or HSD17B2 (17β-HSD2). As such, they are believed to be...
Imhotex patents new NOD2 activators for Crohn’s disease
Patents
Imhotex Ltd. has disclosed desmuramylpeptide (DMP) analogues of muramyl dipeptide (MDP) acting as nucleotide-binding oligomerization domain-containing protein 2 (NOD2) activators reported to be useful for the treatment of Crohn’s disease.
Numab and Kaken sign agreement for NM-81 for IBD
Collaboration
Numab Therapeutics AG and Kaken Pharmaceutical Co. Ltd. have entered into a strategic licensing and co-development agreement for NM-81, a multi-specific antibody for the treatment of inflammatory bowel disease (IBD).

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